Favourable-histology Wilms' tumours are associated with very high 5-year overall survival rates of >90%.[107]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007 Dec;13(4):463-70.
http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com
[16]National Cancer Institute. Statistics for cancers in children, adolescents, and young adults. Oct 2024 [internet publication].
https://nccrexplorer.ccdi.cancer.gov
[2]Espinoza AF, Onwuka E, Siegel DA, et al. Incidence and survival of children and adolescents with Wilms tumor, United States, 2001-2020. Cancer Med. 2025 Feb;14(3):e70598.
https://onlinelibrary.wiley.com/doi/10.1002/cam4.70598
http://www.ncbi.nlm.nih.gov/pubmed/39928531?tool=bestpractice.com
However, there is an increased risk of treatment-related morbidity and mortality 25 years from diagnosis.[130]Termuhlen AM, Tersak JM, Liu Q, et al. Twenty-five year follow-up of childhood Wilms tumor: a report from the Childhood Cancer Survivor Study. Pediatr Blood Cancer. 2011 Dec 15;57(7):1210-6.
http://www.ncbi.nlm.nih.gov/pubmed/21384541?tool=bestpractice.com
Patients with tumour weight <550 g and age <2 years, in particular, have excellent outcomes and may be offered nephrectomy-only followed by imaging surveillance (according to the Children's Oncology Group [COG] treatment approach). These patients have a rate of cure near 90% with surgery alone and avoid the risk of adverse effects from adjuvant chemotherapy.[98]Fernandez CV, Perlman EJ, Mullen EA, et al. Clinical outcome and biological predictors of relapse after nephrectomy only for very low-risk Wilms tumor: a report from Children's Oncology Group AREN0532. Ann Surg. 2017 Apr;265(4):835-40.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5145762
http://www.ncbi.nlm.nih.gov/pubmed/27811504?tool=bestpractice.com
[131]Green DM. The treatment of stages I-IV favorable histology Wilms' tumor. J Clin Oncol. 2004 Apr 15;22(8):1366-72.
http://www.ncbi.nlm.nih.gov/pubmed/15084612?tool=bestpractice.com
[132]Breslow NE, Beckwith JB, Haase GM, et al. Radiation therapy for favorable histology Wilms tumor: prevention of flank recurrence did not improve survival on National Wilms Tumor Studies 3 and 4. Int J Radiat Oncol Biol Phys. 2006 May 1;65(1):203-9.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1483841
http://www.ncbi.nlm.nih.gov/pubmed/16542795?tool=bestpractice.com
[133]Shamberger RC, Anderson JR, Breslow NE, et al. Long-term outcomes for infants with very low risk Wilms tumor treated with surgery alone in National Wilms Tumor Study-5. Ann Surg. 2010 Mar;251(3):555-8.
http://www.ncbi.nlm.nih.gov/pubmed/20142733?tool=bestpractice.com
Presence of anaplasia, aneuploidy, distant metastatic disease (especially to the lymph node and/or hepatic metastasis and/or intravascular tumour thrombus), and loss of heterozygosity (LOH) at 1p and 16q, or gain of chromosome 1q in the tumour genetic profile confers poor prognosis.[32]Gratias EJ, Jennings LJ, Anderson JR, et al. Gain of 1q is associated with inferior event-free and overall survival in patients with favorable histology Wilms tumor: a report from the Children's Oncology Group. Cancer. 2013 Nov 1;119(21):3887-94.
http://www.ncbi.nlm.nih.gov/pubmed/23983061?tool=bestpractice.com
[33]Grundy PE, Breslow NE, Li S, et al. Loss of heterozygosity for chromosomes 1p and 16q is an adverse prognostic factor in favorable-histology Wilms tumor: a report from the National Wilms Tumor Study Group. J Clin Oncol. 2005 Oct 10;23(29):7312-21.
http://www.ncbi.nlm.nih.gov/pubmed/16129848?tool=bestpractice.com
[58]Wittmann S, Zirn B, Alkassar M, et al. Loss of 11q and 16q in Wilms tumors is associated with anaplasia, tumor recurrence, and poor prognosis. Genes Chromosomes Cancer. 2007 Feb;46(2):163-70.
http://www.ncbi.nlm.nih.gov/pubmed/17099873?tool=bestpractice.com
[59]Gratias EJ, Dome JS, Jennings LJ, et al. Association of chromosome 1q gain with inferior survival in favorable-histology Wilms tumor: a report from the Children's Oncology Group. J Clin Oncol. 2016 Sep 10;34(26):3189-94.
https://ascopubs.org/doi/10.1200/JCO.2015.66.1140?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
http://www.ncbi.nlm.nih.gov/pubmed/27400937?tool=bestpractice.com
[79]Skotnicka-Klonowicz G, Rieske P, Bartkowiak J, et al. 16q heterozygosity loss in Wilms' tumour in children and its clinical importance. Eur J Surg Oncol. 2000 Feb;26(1):61-6.
http://www.ncbi.nlm.nih.gov/pubmed/10718182?tool=bestpractice.com
[107]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007 Dec;13(4):463-70.
http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com
[117]Dome JS, Cotton CA, Perlman EJ, et al. Treatment of anaplastic histology Wilms' tumor: results from the fifth National Wilms' Tumor Study. J Clin Oncol. 2006 May 20;24(15):2352-8.
http://www.ncbi.nlm.nih.gov/pubmed/16710034?tool=bestpractice.com
[123]Malogolowkin M, Cotton CA, Green DM, et al. Treatment of Wilms tumor relapsing after initial treatment with vincristine, actinomycin D, and doxorubicin. A report from the National Wilms Tumor Study Group. Pediatr Blood Cancer. 2008 Feb;50(2):236-41.
http://www.ncbi.nlm.nih.gov/pubmed/17539021?tool=bestpractice.com
[134]Osterheld MC, Caron L, Meagher-Villemure K. Role of DNA content analysis and immunohistochemistry in the evaluation of the risk of unfavourable outcome in Wilms' tumours. Anticancer Res. 2008 Mar-Apr;28(2A):751-6.
http://www.ncbi.nlm.nih.gov/pubmed/18507016?tool=bestpractice.com
[135]Nakadate H, Yokomori K, Watanabe N, et al. Mutations/deletions of the WT1 gene, loss of heterozygosity on chromosome arms 11p and 11q, chromosome ploidy and histology in Wilms' tumors in Japan. Int J Cancer. 2001 Nov 1;94(3):396-400.
http://onlinelibrary.wiley.com/doi/10.1002/ijc.1475/full
http://www.ncbi.nlm.nih.gov/pubmed/11745420?tool=bestpractice.com
[136]Messahel B, Williams R, Ridolfi A, et al. Allele loss at 16q defines poorer prognosis Wilms tumour irrespective of treatment approach in the UKW1-3 clinical trials: a Children's Cancer and Leukaemia Group (CCLG) study. Eur J Cancer. 2009 Mar;45(5):819-26.
http://www.ncbi.nlm.nih.gov/pubmed/19231157?tool=bestpractice.com
[137]Chagtai T, Zill C, Dainese L, et al. Gain of 1q as a prognostic biomarker in Wilms tumors (WTs) treated with preoperative chemotherapy in the International Society of Paediatric Oncology (SIOP) WT 2001 trial: a SIOP renal tumours biology consortium study. J Clin Oncol. 2016 Sep 10;34(26):3195-203.
https://ascopubs.org/doi/10.1200/JCO.2015.66.0001?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
http://www.ncbi.nlm.nih.gov/pubmed/27432915?tool=bestpractice.com
Lymph-node involvement and the presence of microscopic residual disease are highly predictive of event-free survival and overall survival in patients with stage III favourable-histology disease.[138]Ehrlich PF, Anderson JR, Ritchey ML, et al. Clinicopathologic findings predictive of relapse in children with stage III favorable-histology Wilms tumor. J Clin Oncol. 2013 Mar 20;31(9):1196-201.
http://www.ncbi.nlm.nih.gov/pubmed/23382471?tool=bestpractice.com
Prognosis is considered excellent in patients with stage IV disease if histology is favourable, metastatic disease is isolated to either the liver or lungs, and metastases responds to treatment with preoperative chemotherapy.[139]Berger M, Fernandez-Pineda I, Cabello R, et al. The relationship between the site of metastases and outcome in children with stage IV Wilms tumor: data from 3 European pediatric cancer institutions. J Pediatr Hematol Oncol. 2013 Oct;35(7):518-24.
http://www.ncbi.nlm.nih.gov/pubmed/23588334?tool=bestpractice.com
Worse outcomes are reported in patients with extra-pulmonary metastases from favourable histology Wilms' tumour compared to those with lung-only metastases.[106]Benedetti DJ, Varela CR, Renfro LA, et al. Treatment of children with favorable histology Wilms tumor with extrapulmonary metastases: a report from the COG studies AREN0533 and AREN03B2 and NWTSG study NWTS-5. Cancer. 2024 Mar 15;130(6):947-61.
https://acsjournals.onlinelibrary.wiley.com/doi/10.1002/cncr.35099
http://www.ncbi.nlm.nih.gov/pubmed/37933882?tool=bestpractice.com
Prognosis is inferior for patients with high-risk stage IV tumours; the overall 5-year survival rates are 17% for blastemal type stage IV Wilms' tumour and 16% for diffuse anaplastic stage IV Wilms' tumour.[140]Pasqualini C, Furtwängler R, van Tinteren H, et al. Outcome of patients with stage IV high-risk Wilms tumour treated according to the SIOP2001 protocol: a report of the SIOP Renal Tumour Study Group. Eur J Cancer. 2020 Mar;128:38-46.
http://www.ncbi.nlm.nih.gov/pubmed/32109849?tool=bestpractice.com
Recurrence rates are low.[107]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007 Dec;13(4):463-70.
http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com
Adverse prognostic factors for patients with recurrent tumours include previous treatment with doxorubicin, relapses that occur <12 months after diagnosis and intra-abdominal relapse after abdominal irradiation.[107]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007 Dec;13(4):463-70.
http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com
[121]Speafico F, Pritchard Jones K, Malogolowkin MH, et al. Treatment of relapsed Wilms tumors: lessons learned. Expert Rev Anticancer Ther. 2009 Dec;9(12):1807-15.
http://www.ncbi.nlm.nih.gov/pubmed/19954292?tool=bestpractice.com
[141]Grundy P, Breslow N, Green DM, et al. Prognostic factors for children with recurrent Wilms' tumor: results from the Second and Third National Wilms' Tumor Study. J Clin Oncol. 1989 May;7(5):638-47.
http://www.ncbi.nlm.nih.gov/pubmed/2540289?tool=bestpractice.com
Prognosis for patients with recurrence is poor.
Poor outcomes are observed in patients with Denys-Drash syndrome as the disease is often bilateral and associated with progressive renal failure.[63]Breslow NE, Collins AJ, Ritchey ML, et al. End stage renal disease in patients with Wilms tumor: results from the National Wilms Tumor Study Group and the United States Renal Data System. J Urol. 2005 Nov;174(5):1972-5.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1483840
http://www.ncbi.nlm.nih.gov/pubmed/16217371?tool=bestpractice.com
[101]Heppe RK, Koyle MA, Beckwith JB. Nephrogenic rests in Wilms tumor patients with the Drash syndrome. J Urol. 1991 Jun;145(6):1225-8.
http://www.ncbi.nlm.nih.gov/pubmed/1851891?tool=bestpractice.com
[142]Breslow NE, Takashima JR, Ritchey ML, et al. Renal failure in the Denys-Drash and Wilms tumor-aniridia syndromes. Cancer Res. 2000 Aug 1;60(15):4030-2.
http://cancerres.aacrjournals.org/cgi/content/full/60/15/4030
http://www.ncbi.nlm.nih.gov/pubmed/10945603?tool=bestpractice.com
However, overall survival is improved with successful renal transplant.[143]Kist-van Holthe JE, Ho PL, Stablein D, et al. Outcome of renal transplantation for Wilms' tumor and Denys-Drash syndrome: a report of the North American Pediatric Renal Transplant Cooperative Study. Pediatr Transplant. 2005 Jun;9(3):305-10.
http://www.ncbi.nlm.nih.gov/pubmed/15910385?tool=bestpractice.com
One study found that Hispanic ethnicity may be associated with poorer prognosis; however, further research on ethnic disparities in Wilms' tumour is required.[3]Lovvorn HN 3rd, Renfro LA, Benedetti DJ, et al. Race and ethnic group enrollment and outcomes for Wilms tumor: analysis of the current era children's oncology group study, AREN03B2. J Am Coll Surg. 2024 Apr 1;238(4):733-49.
http://www.ncbi.nlm.nih.gov/pubmed/38251681?tool=bestpractice.com
[144]Amirian ES. The role of Hispanic ethnicity in pediatric Wilms' tumor survival. Pediatr Hematol Oncol. 2013 May;30(4):317-27.
http://www.ncbi.nlm.nih.gov/pubmed/23484868?tool=bestpractice.com